A Multidimensional Rasch Analysis of the Functional Independence Measure Based on the National Institute on Disability, Independent Living, and Rehabilitation Research Traumatic Brain Injury Model Systems National Database.

A number of studies have evaluated the psychometric properties of the Functional Independence Measure (FIM™) using Rasch analysis, although none has done so using the National Institute on Disability, Independent Living, and Rehabilitation Research Traumatic Brain Injury Model Systems National Database, a longitudinal database that captures demographic and outcome information on persons with moderate to severe traumatic brain injury across the United States. In the current study, we examine the psychometric properties of the FIM as represented by persons within this database and demonstrate that the FIM comprises three subscales representing cognitive, self-care, and mobility domains. These subscales were analyzed simultaneously using a multivariate Rasch model in combination with a time dependent concurrent calibration scheme with the goal of creating a raw score-to-logit transformation that can be used to improve the accuracy of parametric statistical analyses. The bowel and bladder function items were removed because of misfit with the motor and cognitive items. Some motor items exhibited step disorder, which was addressed by collapsing Categories 1-3 for Toileting, Stairs, Locomotion, Tub/Shower Transfers; Categories 1 and 2 for Toilet and Bed Transfers; and Categories 2 and 3 for Grooming. The strong correlations (r = 0.82-0.96) among the three subscales suggest they should be modeled together. Coefficient alpha of 0.98 indicates high internal consistency. Keyform maps are provided to enhance clinical interpretation and application of study results.

Myotonic dystrophy health index: Correlations with clinical tests and patient function.

INTRODUCTION: The Myotonic Dystrophy Health Index (MDHI) is a disease-specific patient-reported outcome measure. Here, we examine the associations between the MDHI and other measures of disease burden in a cohort of individuals with myotonic dystrophy type-1 (DM1). METHODS: We conducted a cross-sectional study of 70 patients with DM1. We examined the associations between MDHI total and subscale scores and scores from other clinical tests. Participants completed assessments of strength, myotonia, motor and respiratory function, ambulation, and body composition. Participants also provided blood samples, underwent physician evaluations, and completed other patient-reported outcome measures. RESULTS: MDHI total and subscale scores were strongly associated with muscle strength, myotonia, motor function, and other clinical measures. CONCLUSIONS: Patient-reported health status, as measured by the MDHI, is associated with alternative measures of clinical health. These results support the use of the MDHI as a valid tool to measure disease burden in DM1 patients.

Patient-Reported Impact of Symptoms in Myotonic Dystrophy Type 2 (PRISM-2).

OBJECTIVE: To determine the frequency and relative importance of the most life-affecting symptoms in myotonic dystrophy type 2 (DM2) and to identify the factors that have the strongest association with these symptoms. METHODS: We conducted a cross-sectional study of adult patients with DM2 from a National Registry of DM2 Patients to assess the prevalence and relative importance of 310 symptoms and 21 symptomatic themes. Participant responses were compared by age categories, sex, educational attainment, employment status, and duration of symptoms. RESULTS: The symptomatic themes with the highest prevalence in DM2 were the inability to do activities (94.4%), limitations with mobility or walking (89.2%), hip, thigh, or knee weakness (89.2%), fatigue (89.2%), and myotonia (82.6%). Participants identified the inability to do activities and fatigue as the symptomatic themes that have the greatest overall effect on their lives. Unemployment, a longer duration of symptoms, and less education were associated with a higher average prevalence of all symptomatic themes (p < 0.01). Unemployment, a longer duration of symptoms, sex, and increased age were associated with a higher average effect of all symptomatic themes among patients with DM2 (p < 0.01). CONCLUSIONS: The lives of patients with DM2 are affected by a variety of symptoms. These symptoms have different levels of significance and prevalence in this population and vary across DM2 subgroups in different demographic categories.

New English and Spanish social health measures will facilitate evaluating health determinants.

OBJECTIVE: To develop psychometrically sound, culturally relevant, and linguistically equivalent English and Spanish self-report measures of social health guided by a comprehensive conceptual model and applicable across chronic illnesses. METHODS: The Patient-Reported Outcomes Measurement Information System (PROMIS) Social Health Workgroup implemented a mixed methods approach to evaluate earlier results (v1.0); expand and refine domain definitions and items; translate items into Spanish; and obtain qualitative feedback. Computer-based and paper/pencil questionnaire administration was conducted with a variety of U.S. respondent samples during 2009-2012. Analyses included exploratory factor analysis (EFA), confirmatory factor analysis (CFA), two-parameter logistic item response theory (IRT) modeling, evaluation of differential item functioning (DIF), and evaluation of criterion and construct validity. RESULTS: Qualitative feedback supported the conceptualization of the Social Health domain framework (Social Function and Social Relationships subcomponents). Validation testing participants (n = 2,208 English; n = 644 Spanish) were diverse in terms of gender, age, education, and ethnicity/race. EFA, CFA, and IRT identified 7 unidimensional factors with good model fit. There was no DIF by language, and good evidence of criterion and construct validity. CONCLUSIONS: PROMIS English and Spanish language instruments (v2.0), including computer-adaptive tests and fixed-length short forms, are publicly available for assessment of Social Function (Ability to Participate in Social Roles and Activities, and Satisfaction with Social Roles and Activities) and Social Relationships (Companionship; Emotional, Informational and Instrumental Support; and Social Isolation). Measures of social health will play a key role in applications that use ecologic (or determinants of health) models that emphasize how patients' social environments influence their health.

Ensuring comprehensive assessment of urinary problems in prostate cancer through patient-physician concordance.

OBJECTIVES: To examine the concordance between clinicians and men diagnosed with prostate cancer on a clinician-derived pathophysiological classification of the following self-reported urinary complications: storage (irritative), voiding (obstructive), and leakage/incontinence. MATERIALS AND METHODS: Fourteen urology experts classified 37 urinary function questionnaire items into 3 primary conceptual dimensions (e.g., storage [irritative], voiding [obstructive] and urinary leakage/incontinence) that would best reflect each item's content. In addition, 218 patient participants provided responses to the 37 items. Using classifications by experts to develop the conceptual framework, the structure was tested using confirmatory factor analyses with patient data. RESULTS: Expert consensus was achieved in the classification of 31 out of 37 items. Using the 3-factor conceptual framework and patient data, the fit indices for the overall correlated factor model suggested an acceptable overall model fit. The analyses of the separate domains showed acceptable fit for the storage/irritative domain and the leaking/incontinence domain. The dimensionality of the voiding/obstructive domain was too difficult to estimate. CONCLUSIONS: Our analysis found items that conceptually and psychometrically support 2 constructs (leaking/incontinence and storage/irritative). The consistency of this support between the groups suggests a clinical relevance that is useful in treating patients. We have conceptual support for a third hypothesis (voiding/obstructive), although there were too few items to assess this psychometrically. Relative motivating factors of bother and urinary complaints were not addressed and remain an unmet need in this field.

Self-scoring templates for motor and cognitive subscales of the FIM instrument for persons with spinal cord injury.

OBJECTIVE: To provide self-scoring templates for the FIM instrument's motor and cognitive scales that enable clinicians to monitor progress during rehabilitation using equal-interval Rasch-calibrated measures instead of ordinal raw scores. DESIGN: Secondary analysis of a prospective, observational cohort study. SETTING: Six geographically dispersed hospital-based rehabilitation centers in the United States. PARTICIPANTS: Subset of consecutively enrolled individuals with new traumatic spinal cord injuries discharged from participating rehabilitation centers (N=1146). INTERVENTION: Not applicable. MAIN OUTCOME MEASURES: Subscores of the FIM instrument, including a 13-item motor scale, a 5-item cognitive scale, an 11-item (without sphincter control items) motor scale, a 3-item transfer scale, a 6-item self-care scale, a 3-item self-care upper extremity scale, and a 3-item self-care lower extremity scale. RESULTS: KeyForms for the FIM instrument scales allow clinicians and investigators to estimate patients' functional status and monitor progress. In cases with no missing data, the look-up tables provide more accurate estimates of patients' functional status. CONCLUSION: Clinicians can use KeyForms and look-up tables for FIM instrument subscales to monitor patients' progress and communicate improvement in equal-interval units.

Myotonic Dystrophy Health Index: initial evaluation of a disease-specific outcome measure.

INTRODUCTION: In preparation for clinical trials we examine the validity, reliability, and patient understanding of the Myotonic Dystrophy Health Index (MDHI). METHODS: Initially we partnered with 278 myotonic dystrophy type-1 (DM1) patients and identified the most relevant questions for the MDHI. Next, we used factor analysis, patient interviews, and test-retest reliability assessments to refine and evaluate the instrument. Lastly, we determined the capability of the MDHI to differentiate between known groups of DM1 participants. RESULTS: Questions in the final MDHI represent 17 areas of DM1 health. The internal consistency was acceptable in all subscales. The MDHI had a high test-retest reliability (ICC = 0.95) and differentiated between DM1 patient groups with different disease severities. CONCLUSIONS: Initial evaluation of the MDHI provides evidence that it is valid and reliable as an outcome measure for assessing patient-reported health. These results suggest that important aspects of DM1 health may be measured effectively using the MDHI.

Development and validation of the functional assessment of chronic illness therapy treatment satisfaction (FACIT TS) measures.

PURPOSE: To develop and validate a new functional assessment of chronic illness therapy (FACIT) measure of satisfaction with treatment for chronic illnesses such as cancer and HIV/AIDS. METHODS: To define domains and generate items, a literature review informed creation of semi-structured interview guides for patients and an international expert panel of clinicians and researchers. Patients and experts also rated 15 areas of satisfaction for relevance. The final list of items underwent further refinement by the original expert panel and a new group of clinical experts. Items were tested in four studies (primarily lung cancer) and data were pooled for analysis. Exploratory and confirmatory factor analyses (CFA), and item response theory modeling were conducted to evaluate dimensionality. Internal consistency reliability and test-retest reliability were both evaluated. Validity was evaluated by correlating the FACIT subscale scores and measures of comparable concepts and by testing the scales' ability to distinguish people according to their overall treatment satisfaction. RESULTS: Two instruments were created: the FACIT TS-general (G), an overall evaluation of current treatment, and the FACIT TS-patient satisfaction (PS), a measure of patient satisfaction. CFA results were not optimal for a five-factor solution for PS. Internal consistency reliability met psychometric standards (≥0.70) for all PS subscales. Construct validity was established for the PS subscales: Physician Communication, Treatment Staff Communication, Technical Competence, Confidence and Trust, and Nurse Communication. CONCLUSIONS: The two instruments generated here offer a new way to assess several key dimensions of patient satisfaction with treatment, especially for people with lung cancer.

Measuring enfranchisement: importance of and control over participation by people with disabilities.

OBJECTIVE: To evaluate the psychometric properties and validity of an expanded set of community enfranchisement items that are suitable for computer adaptive testing. DESIGN: Survey. SETTING: Community setting. PARTICIPANTS: Individuals with disabilities (N=1163) were recruited from an online panel generation company (51%), former rehabilitation inpatients (18%), disability community organizations (13%), a registry of rehabilitation patients (10%), and Traumatic Brain Injury and Spinal Cord Injury Model System facilities (8%). Inclusion criteria were a self-identified disability, aged ≥18 years, and the ability to read and speak English. INTERVENTIONS: None. MAIN OUTCOME MEASURE: Community enfranchisement. RESULTS: Exploratory and confirmatory factor analyses of the 48 enfranchisement items suggested 2 distinct subsets of items: (1) importance of participation and (2) control over participation. Principal components analysis of the residuals suggested that the 2 item sets are unidimensional. Rating scale analysis provided evidence that the 2 item sets fit the Rasch model. Importance and control were moderately correlated with each other and with disability severity. CONCLUSIONS: Importance of participation and control over participation define 2 distinct sets of participation enfranchisement. Preliminary evidence supports their validity.

Emotion assessment using the NIH Toolbox.

One of the goals of the NIH Toolbox for Assessment of Neurological and Behavioral Function was to identify or develop brief measures of emotion for use in prospective epidemiologic and clinical research. Emotional health has significant links to physical health and exerts a powerful effect on perceptions of life quality. Based on an extensive literature review and expert input, the Emotion team identified 4 central subdomains: Negative Affect, Psychological Well-Being, Stress and Self-Efficacy, and Social Relationships. A subsequent psychometric review identified several existing self-report and proxy measures of these subdomains with measurement characteristics that met the NIH Toolbox criteria. In cases where adequate measures did not exist, robust item banks were developed to assess concepts of interest. A population-weighted sample was recruited by an online survey panel to provide initial item calibration and measure validation data. Participants aged 8 to 85 years completed self-report measures whereas parents/guardians responded for children aged 3 to 12 years. Data were analyzed using a combination of classic test theory and item response theory methods, yielding efficient measures of emotional health concepts. An overview of the development of the NIH Toolbox Emotion battery is presented along with preliminary results. Norming activities led to further refinement of the battery, thus enhancing the robustness of emotional health measurement for researchers using the NIH Toolbox.

Assessing social support, companionship, and distress: National Institute of Health (NIH) Toolbox Adult Social Relationship Scales.

OBJECTIVE: The quality of our daily social interactions-including perceptions of support, feelings of loneliness, and distress stemming from negative social exchanges-influence physical health and well-being. Despite the importance of social relationships, brief yet precise unidimensional scales that assess key aspects of social relationship quality are lacking. As part of the National Institute of Health (NIH) Toolbox for the Assessment of Neurological and Behavioral Function, we developed brief self-report scales designed to assess aspects of social support, companionship, and social distress across age cohorts. This article details the development and psychometric testing of the adult NIH Toolbox Social Relationship scales. METHODS: Social relationship concepts were selected, and item sets were developed and revised based on expert feedback and literature review. Items were then tested across a community-dwelling U.S. Internet panel sample of adults aged 18 and above (N = 692) using traditional (classic) psychometric methods and item response theory approaches to identify items for inclusion in 5-8 item unidimensional scales. Finally, concurrent validity of the newly developed scales was evaluated with respect to their interrelationships with classic social relationship validation instruments. RESULTS: Results provide support for the internal reliability and concurrent validity of resulting self-report scales assessing Emotional Support, Instrumental Support, Friendship, Loneliness, Perceived Rejection, and Perceived Hostility. CONCLUSION: These brief social relationship scales provide the pragmatic utility and enhanced precision needed to promote future epidemiological and social neuroscience research on the impact of social relationships on physical and emotional health outcomes.

Patient-reported impact of symptoms in myotonic dystrophy type 1 (PRISM-1).

OBJECTIVE: To determine the most critical symptoms in a national myotonic dystrophy type 1 (DM1) population and to identify the modifying factors that have the greatest effect on the severity of these symptoms. METHODS: We performed a cross-sectional study of 278 adult patients with DM1 from the national registry of patients with DM1 between April and August 2010. We assessed the prevalence and relative significance of 221 critical DM1 symptoms and 14 disease themes. These symptoms and themes were chosen for evaluation based on prior interviews with patients with DM1. Responses were categorized by age, CTG repeat length, gender, and duration of symptoms. RESULTS: Participants with DM1 provided symptom rating survey responses to address the relative frequency and importance of each DM1 symptom. The symptomatic themes with the highest prevalence in DM1 were problems with hands or arms (93.5%), fatigue (90.8%), myotonia (90.3%), and impaired sleep or daytime sleepiness (87.9%). Participants identified fatigue and limitations in mobility as the symptomatic themes that have the greatest effect on their lives. We found an association between age and the average prevalence of all themes (p < 0.01) and between CTG repeat length and the average effect of all symptomatic themes on participant lives (p < 0.01). CONCLUSIONS: There are a wide range of symptoms that significantly affect the lives of patients with DM1. These symptoms, some previously underrecognized, have varying levels of importance in the DM1 population and are nonlinearly dependent on patient age and CTG repeat length.

Developing an emotional distress item bank for cancer patients.

Emotional distress is common among cancer patients during and after treatment. Many instruments have been used to measure emotional distress; however, none of them has emerged as a standard. Although the diversity of instruments has some merit, the lack of a common measure limits our ability to compare studies. This paper describes how we constructed a 46-item emotional distress bank. Using expert judgment, we selected a pool of items with emotional content from this six-instrument set. Rasch rating scale analysis helped us identify a set of general distress items with good model fit and a measurement gap causing floor effects. Additional items were written to augment the measure where found deficient. The resulting set of items reflects a spectrum of positive and negative affect. The measure demonstrated excellent reliability (person separation reliability = .96) and a wide range of emotional distress and was able to distinguish among levels of disease severity.

The symptom inventory disability-specific short forms for multiple sclerosis: construct validity, responsiveness, and interpretation.

OBJECTIVES: To test the cross-sectional and longitudinal construct validity of the disability-specific short forms of the Symptom Inventory for multiple sclerosis, to compare its internal consistency reliability and construct validity with those of the original (1999) 29-item short form of the Symptom Inventory, and to provide for the new disability-specific short forms interpretation metrics for use in sample size calculation for future research. DESIGN: A Web-based longitudinal study, with data collected at baseline and 6 months after baseline. Correlations evaluated the overlap among disease-specific and generic patient-reported outcome measures. Responsiveness was assessed by using symptom transition scores and modified standardized response means. Interpretation guidelines were provided by using Cohen's effect size and crosswalks to the disease-specific and generic quality-of-life measures. SETTING: National Multiple Sclerosis Registry. PARTICIPANTS: People with multiple sclerosis (N=1142) who participated in the North American Research Committee on Multiple Sclerosis Registry. INTERVENTIONS: Not applicable. MAIN OUTCOME MEASURES: The Symptom Inventory; the disease-specific Performance Scales and the Patient-Determined Disease Steps; the generic Short Form 12. RESULTS: The Symptom Inventory evidenced convergent and divergent validity, and the disability-specific short forms evidenced similar psychometric performance as the 1999 short form but had slightly better alpha reliability. They also evidenced moderate responsiveness to clinically important change, with more responsiveness among those with mild and moderate disabilities than among those with severe disabilities. Effect sizes were larger among patients who reported symptom improvement, rather than deterioration, suggesting that the tool would be more useful in clinical research focused on testing whether an intervention improves symptom experience, particularly for patients with mild and moderate disabilities. Crosswalks provided graphic and numeric links between the Symptom Inventory and other patient-reported outcomes. CONCLUSIONS: The Symptom Inventory can be useful for elucidating the patient's experience, but it varies considerably across and within disability groupings. Directions for future research are discussed.

Development of a measure of skin care belief scales for persons with spinal cord injury.

OBJECTIVES: To develop and validate a measure of skin care beliefs and to describe the skin care behaviors of persons with spinal cord injury (SCI). DESIGN: A mixed-methods design was used to develop the Skin Care Beliefs Scales (SCBS). The health belief model framed the hypotheses. Phase 1 included item development, content validity testing, and pilot testing. Phase 2 included testing the scale structure (principal components analysis), internal consistency reliability, test-retest reliability, and relationships between the belief scales and care behaviors. SETTING: Two acute rehabilitation hospitals and Internet websites. PARTICIPANTS: Patients with SCI (N=462; qualitative/pilot n=56; psychometric study n=406) participated. INTERVENTIONS: Not applicable. MAIN OUTCOME MEASURES: The pilot and phase 2 studies, respectively, used 146-item and 114-item versions of the SCBS. A skin care activity log was used to record skin care behaviors. RESULTS: Content validity indicated that the items were relevant and clear. The analysis resulted in 11 independent scales reflecting 3 general beliefs (susceptibility, severity, self-efficacy) and barrier and benefit behavior-specific scales for skin checks, wheelchair pressure reliefs, and turning and sitting times. With the exception of skin check barriers (α=.65), Cronbach alphas of the scale ranged from .74 to .94. Test-retest intraclass correlations were fair to excellent (range, .42-.75). Construct validity was supported. Hierarchical linear regression indicated that turning benefits, barriers, susceptibility, and self-efficacy were significant predictors of turning time. Benefits or barriers were correlated significantly with skin check and pressure relief adherence (ρ range, -.17 to -.33). Self-efficacy was correlated with wheelchair pressure relief (ρ=.18). Skin care behavior adherence varied widely (eg, 0%-100%). CONCLUSIONS: The scales showed acceptable reliability and validity. Further testing with larger samples is desirable.

The symptom inventory disability-specific short forms for multiple sclerosis: reliability and factor structure.

OBJECTIVE: To further the development of the 99-item Symptom Inventory (SI) for multiple sclerosis (MS) using modern test theory methods to create 3 disability-specific short forms for MS patient subgroups identified using Performance Scale (PS) items. DESIGN: A web-based cross-sectional study. SETTING: National MS Registry. PARTICIPANTS: People with MS (N=1532) who participate in the North American Research Committee on Multiple Sclerosis Registry. INTERVENTIONS: None. MAIN OUTCOME MEASURES: The SI; the disease-specific PS and the Patient-Determined Disease Steps; and the generic Short-Form 12. RESULTS: When the original SI subscales did not demonstrate unidimensionality, exploratory factor analysis was conducted yielding 14 factors that could be classified using the structure of the PS. Confirmatory factor analysis confirmed the unidimensionality of the hand function, vision, fatigue, cognitive, bowel/bladder, spasticity, and pain scales. The mobility scale was split into mobility and use of assistive devices; the sensory scale was split into sensory and vasomotor. Item response theory analyses revealed good model fit. CONCLUSIONS: This study provides empirical support for a 10-scale symptom measure for use in MS clinical research, with short forms in 5 scales tailored to have good specificity for people with mild, moderate, and severe disability and single forms for the remaining 5 scales. The PS items can serve as a screener for these disability-specific short forms, which provide choice and flexibility that are similar to a computerized adaptive test but without the reliance on real-time computer infrastructure.

Neuro-QOL: quality of life item banks for adults with neurological disorders: item development and calibrations based upon clinical and general population testing.

PURPOSE: Neuro-QOL provides a clinically relevant and psychometrically robust health-related quality of life (HRQL) assessment tool for both adults and children with common neurological disorders. We now report the psychometric results for the adult tools. METHODS: An extensive research, survey and consensus process was used to produce a list of 5 priority adult neurological conditions (stroke, multiple sclerosis, Parkinson's disease, epilepsy and ALS). We identified relevant health related quality of life (HRQL) domains through multiple methods and data sources including a comprehensive review of the literature and literature search, expert interviews and surveys and patient and caregiver focus groups. The final domain framework consisted of 17 domains of Physical, Mental and Social health. There were five phases of item development: (1) identification of 3,482 extant items, (2) item classification and selection, (3) item review and revision, (4) cognitive interviews with 63 patients to assess their understanding of individual items and (5) field testing of 432 representative items. PARTICIPANTS AND PROCEDURES: Participants were drawn from the US general population and clinical settings, and included both English and Spanish speaking subjects (N = 3,246). Confirmatory factor analysis (CFA) was used to evaluate the dimensionality of unidimensional domains. Where the domain structure was previously unknown, the dataset was split and first analyzed with exploratory factor analysis and then CFA. Samejima's graded response model (GRM) was used to calculate IRT parameters. We further evaluated differential item functioning (DIF) on gender, education and age. RESULTS: Thirteen unidimensional calibrated item banks consisting of 297 items were developed. All of the tested item banks had high reliability and few or no locally dependent items. The range of item slopes and thresholds with good information are reported for each of the item banks. The banks can support CAT and the development of short forms. CONCLUSION: The Neuro-QOL measurement system provides item banks and short forms that enable PRO measurement in neurological research, minimizes patient burden and can be used to create multiple instrument types minimizing standard error. The 17 adult measures include 13 calibrated item banks, 3 item pools available for calibration work by others, and 1 stand-alone scale (index). The Neuro-QOL instruments provide a "common metric" of representative concepts for use across patient groups in different studies.

Acupuncture for treatment of insomnia in patients with traumatic brain injury: a pilot intervention study.

OBJECTIVES: : To assess the efficacy of acupuncture in treating insomnia in traumatic brain injury (TBI) survivors as compared to medication, to determine whether acupuncture has fewer cognitive and affective adverse effects than does medication. PARTICIPANTS: : Twenty-four adult TBI survivors, randomized to acupuncture or control arms. SETTING: : Outpatient rehabilitation clinic. MEASURES: : Insomnia Severity Index (degree of insomnia); actigraphy (sleep time); Hamilton Depression Rating Scale (depression); Repeatable Battery for the Assessment of Neuropsychological Status and Paced Auditory Serial Addition Test (cognitive function) administered at baseline and postintervention. RESULTS: : Sleep time did not differ between the treatment and control groups after intervention, whereas cognition improved in the former but not the latter. CONCLUSION: : Acupuncture has a beneficial effect on perception of sleep or sleep quality and on cognition in our small sample of patients with TBI. Further studies of this treatment modality are warranted to validate these findings and to explore factors that contribute to treatment efficacy.

Quality-of-life measures in children with neurological conditions: pediatric Neuro-QOL.

BACKGROUND: A comprehensive, reliable, and valid measurement system is needed to monitor changes in children with neurological conditions who experience lifelong functional limitations. OBJECTIVE: This article describes the development and psychometric properties of the pediatric version of the Quality of Life in Neurological Disorders (Neuro-QOL) measurement system. METHODS: The pediatric Neuro-QOL consists of generic and targeted measures. Literature review, focus groups, individual interviews, cognitive interviews of children and consensus meetings were used to identify and finalize relevant domains and item content. Testing was conducted on 1018 children aged 10 to 17 years drawn from the US general population for generic measures and 171 similarly aged children with muscular dystrophy or epilepsy for targeted measures. Dimensionality was evaluated using factor analytic methods. For unidimensional domains, item parameters were estimated using item response theory models. Measures with acceptable fit indices were calibrated as item banks; those without acceptable fit indices were treated as summary scales. RESULTS: Ten measures were developed: 8 generic or targeted banks (anxiety, depression, anger, interaction with peers, fatigue, pain, applied cognition, and stigma) and 2 generic scales (upper and lower extremity function). The banks reliably (r > 0.90) measured 63.2% to 100% of the children tested. CONCLUSIONS: The pediatric Neuro-QOL is a comprehensive measurement system with acceptable psychometric properties that could be used in computerized adaptive testing. The next step is to validate these measures in various clinical populations.

Response shift in patients with multiple sclerosis: an application of three statistical techniques.

OBJECTIVE: With the evolution of theory and methods for detecting recalibration, reprioritization, and reconceptualization response shifts, the time has come to evaluate and compare the current statistical detection techniques. This manuscript presents an overview of a cross-method validation done on the same patient sample. METHODS: Three statistical techniques were used: Structural Equation Modeling, Latent Trajectory Analysis, and Recursive Partitioning and Regression Tree modeling. The study sample (n = 3,008) was drawn from the North American Research Committee on Multiple Sclerosis (NARCOMS) Registry to represent patients soon after diagnosis, classified as having either a self-reported relapsing, progressive, or stable disease trajectory. Patient-reported outcomes included the disease-specific Performance Scales and the Patient-Derived Disease Steps, and the generic SF-12v2 measure. RESULTS: Small response shift effect sizes were detected by all of the methods. Recalibration response shift was detected by Structural Equation Modeling, Recursive Partitioning Regression Tree demonstrated patterns consistent with all three types of response shift, and Latent Trajectory Analysis, although unable to distinguish types of response shift, did detect response shift in less than 1% of the sample. CONCLUSION: The methods and their findings were discussed for operationalization, interpretability, assumptions, ability to use all data points from the study sample, limitations, and strengths. Directions for future research are discussed.